Generalized Lipodystrophy
Conditions
Keywords
Metreleptin
Brief summary
This is an open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or hypertriglyceridaemia
Interventions
DRUGMetreleptin
Metreleptin is a recombinant human leptin analog that is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency
Sponsors
Amryt Pharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Single Group Assignment
Eligibility
Sex/Gender
ALL
Age
No minimum to 5 Years
Healthy volunteers
No
Inclusion criteria
* Confirmed diagnosis of Generalised Lipodystrophy * Metreleptin treatment naive
Exclusion criteria
* Weight \<9 kg at Screening (Visit 1)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent change from baseline in fasting serum TG levels at Month 12 for subjects with fasting TG levels ≥2.3 mmol/L (200 mg/dL) at baseline | 12 months | To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia |
| Absolute change from baseline in glycated haemoglobin (HbA1c) at Month 12 for subjects with HbA1c ≥6.5% at baseline | 12 months | To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of subjects of those with Baseline HbA1c ≥6.5% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <6.5% or HbA1c <5.7% at Month 12 | 12 months | To evaluate the efficacy of metreleptin treatment in patients with GL |
| Proportion of subjects of those with Baseline HbA1c ≥5.7% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <5.7% at Month 12 | 12 months | To evaluate the efficacy of metreleptin treatment in patients with GL |
| Proportion of subjects of those with fasting serum TG ≥ 1.7 mmol/L (150 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12 | 12 months | To evaluate the efficacy of metreleptin treatment in patients with GL |
| Proportion of subjects of those with fasting serum TG ≥ 2.3 mmol/L (200 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12 | 12 months | To evaluate the efficacy of metreleptin treatment in patients with GL |
| Change from baseline in liver volume and liver span as assessed by ultrasound at each post-baseline visit through Month 12 | 12 months | To evaluate the efficacy of metreleptin treatment in patients with GL |
| Incidence of, treatment emergent adverse events (TEAEs), serious adverse events (SAEs), treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation | 12 months | To assess safety and tolerability of metreleptin |
Countries
Belgium, France, Germany, Italy
Outcome results
None listed